Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9004 Administered by Systemic Infusion in Limb Girdle Muscular Dystrophy Type 2D/R3 Participants in the United States

NCTID	NCT06747273 (View at clinicaltrials.gov)
Description	The primary objective of this study is to evaluate the safety of SRP-9004. (Show More)
Development Status	Active
Indication	Limb-Girdle Muscular Dystrophy, Type 2D/R3
Disease Ontology Term	DOID:0110278
Compound Name	SRP-9004
Compound Alias	Patidistrogene bexoparvovec
Compound Description	rAAVrh74-tMCK-SGCA
Sponsor	Sarepta Therapeutics, Inc.
Funder Type	Industry
Recruitment Status	Enrolling by invitation
Enrollment Count	4
Results Posted	Not Available

Target Gene/Variant	SGCA
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAVrh74
Editor Type	none
Dose 1	Phase 1/2 study: 1E12 vg/kg/single limb (n=1)
Dose 2	Phase 1/2 study: 1E12 vg/kg/limb (n=3), total dose: 2E12 vg/kg
Dose 3	Phase 1/2 study: 3E12 vg/kg/limb (n=2), total dose: 6E12 vg/kg
Dose 4
Dose 5

No.of Trial Sites	2
Locations	United States

Has US IND	True
FDA Designations
Recent Updates	Enrollment and dosing is complete